Department of Neurology, Hospital Universitari de Bellvitge, L'Hospitalet de Llobregat, Barcelona, Spain.
Grifols Bioscience Research Group, Grifols, Barcelona, Spain.
Neurol Sci. 2022 May;43(5):3211-3221. doi: 10.1007/s10072-021-05723-z. Epub 2021 Nov 18.
Plasma exchange (PE) is used to treat a range of neurological disorders. Based on results demonstrated in Alzheimer's disease, we theorized that PE with albumin replacement (PE-A) might alter the metabolic profile of plasma and cerebrospinal fluid in patients with amyotrophic lateral sclerosis (ALS) by removing disease-inducing molecules. The aim of this study was to evaluate the effect of PE-A on disease progression in ALS.
In this open-label, non-controlled, single-arm, prospective pilot study, 13 adults with ALS had 6 months' treatment with PE-A 5% and 6 months' follow-up. Primary endpoints were changes from baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score and forced vital capacity (FVC) through 48 weeks. A post hoc analysis compared individual patient data with the expected ALSFRS-R progression slope.
The median ALSFRS-R score declined throughout the study, although the rate of decline was slower than expected in seven patients at treatment end and in five patients at study end. Six patients remained in the same baseline slope progression category, and four patients improved their slope category at treatment end. Median FVC decreased significantly during the study. Treatment was well tolerated. Of 330 PE-A procedures, 0.9% were associated with potentially related adverse events.
Although functional impairment progressed, about two-thirds of patients showed a slower than expected rate of decline at treatment end. Most patients had unaltered (54.5%) or reduced (36.4%) ALSFRS-R slope progression at treatment end. Further evaluation of PE-A in controlled studies involving more patients is warranted.
2013-004842-40.
ClinicalTrials.gov identifier: NCT02479802.
血浆置换（PE）用于治疗多种神经疾病。基于在阿尔茨海默病中得到的结果，我们推测，通过清除致病分子，白蛋白替代的血浆置换（PE-A）可能会改变肌萎缩侧索硬化症（ALS）患者的血浆和脑脊液代谢谱。本研究旨在评估 PE-A 对 ALS 疾病进展的影响。
在这项开放标签、非对照、单臂、前瞻性试验中，13 名成人 ALS 患者接受了 6 个月的 5%PE-A 治疗和 6 个月的随访。主要终点是 48 周时通过基线改变肌萎缩侧索硬化功能评定量表修订版（ALSFRS-R）评分和用力肺活量（FVC）。事后分析比较了个体患者数据与预期的 ALSFRS-R 进展斜率。
尽管在治疗结束时，7 名患者和在研究结束时，5 名患者的 ALSFRS-R 评分下降速度比预期的要慢，但整个研究过程中，中位数 ALSFRS-R 评分一直在下降。6 名患者的基线斜率进展类别保持不变，4 名患者在治疗结束时改善了斜率类别。研究期间 FVC 显著下降。治疗耐受性良好。在 330 次 PE-A 治疗中，有 0.9%与潜在相关的不良事件相关。
尽管功能损伤进展，但约三分之二的患者在治疗结束时表现出比预期更慢的下降速度。大多数患者在治疗结束时 ALSFRS-R 斜率进展没有改变（54.5%）或减少（36.4%）。需要进一步在涉及更多患者的对照研究中评估 PE-A。
2013-004842-40。
ClinicalTrials.gov 标识符：NCT02479802。
